Intellia gets FDA nod to resume gene therapy trial after safety pause

Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a clinical hold on one of its late-stage gene therapy trials, allowing the company to resume testing its experimental drug for a rare nerve disease.
Shares of the drug developer surged over 20% in premarket trading.
The Food and Drug Administration removed the hold on a study testing Intellia’s CRISPR-based therapy, nexiguran ziclumeran, in patients with a hereditary condition ⁠that causes nerve damage. The company said it plans to restart enrolling patients at the soonest.
The drug is designed as a one-time infusion for people with hereditary transthyretin amyloidosis with polyneuropathy, or ATTRv-PN, a disease where a misshapen protein builds up in the nerves and causes progressive damage.
The regulator had paused two related studies in October after a patient in an earlier dosing group developed severe liver related side effects.
Intellia said it has since agreed to stronger safety monitoring measures, which enabled regulators to clear the study to move forward.
A separate study testing the same therapy in ⁠patients whose hearts are affected by the disease remains on hold. Intellia said it is still working with the FDA on that program and would provide an update once a path forward ⁠is agreed upon.
Intellia has expanded the nerve disease trial to enroll about 60 patients, up from the original target ⁠of 50. The study will compare patients who receive the gene therapy against those who get a placebo.